Trial Outcomes & Findings for The Stanford Parkinson's Disease Plasma Study (NCT NCT02968433)
NCT ID: NCT02968433
Last Updated: 2020-12-30
Results Overview
The primary outcome measure is the number of adverse events across all participants that might be related to young plasma infusions as a novel treatment to Parkinson's Disease symptoms. Adverse events were categorized as probably, possibly, or not related to the study intervention.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
16 participants
Primary outcome timeframe
8 weeks
Results posted on
2020-12-30
Participant Flow
Participant milestones
| Measure |
Infusions of Young Plasma
All participants will undergo neuropsychological, neuropsychiatric, and kinematic assessments prior to receiving infusions of young plasma as the treatment.
Participants will receive 1 unit of young plasma, twice a week over a four week duration.
After the four weeks of plasma infusions, participants will undergo neuropsychological, neuropsychiatric and kinematic reassessments. No deception will be used.
Infusions of young plasma: Participants will receive four twice- weekly infusions of 1 unit young plasma (male, ages between 18-25)
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|---|---|
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Overall Study
STARTED
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16
|
|
Overall Study
COMPLETED
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15
|
|
Overall Study
NOT COMPLETED
|
1
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Reasons for withdrawal
| Measure |
Infusions of Young Plasma
All participants will undergo neuropsychological, neuropsychiatric, and kinematic assessments prior to receiving infusions of young plasma as the treatment.
Participants will receive 1 unit of young plasma, twice a week over a four week duration.
After the four weeks of plasma infusions, participants will undergo neuropsychological, neuropsychiatric and kinematic reassessments. No deception will be used.
Infusions of young plasma: Participants will receive four twice- weekly infusions of 1 unit young plasma (male, ages between 18-25)
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|---|---|
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Overall Study
Withdrawal by Subject
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1
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Baseline Characteristics
The Stanford Parkinson's Disease Plasma Study
Baseline characteristics by cohort
| Measure |
Infusions of Young Plasma
n=15 Participants
All participants will undergo neuropsychological, neuropsychiatric, and kinematic assessments prior to receiving infusions of young plasma as the treatment.
Participants will receive 1 unit of young plasma, twice a week over a four week duration.
After the four weeks of plasma infusions, participants will undergo neuropsychological, neuropsychiatric and kinematic reassessments. No deception will be used.
Infusions of young plasma: Participants will receive four twice- weekly infusions of 1 unit young plasma (male, ages between 18-25)
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|---|---|
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Age, Categorical
<=18 years
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0 Participants
n=5 Participants
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Age, Categorical
Between 18 and 65 years
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8 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
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7 Participants
n=5 Participants
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Age, Continuous
|
63 years
STANDARD_DEVIATION 8.3 • n=5 Participants
|
|
Sex: Female, Male
Female
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5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
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10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
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2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=5 Participants
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|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
12 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
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Region of Enrollment
United States
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15 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: 8 weeksThe primary outcome measure is the number of adverse events across all participants that might be related to young plasma infusions as a novel treatment to Parkinson's Disease symptoms. Adverse events were categorized as probably, possibly, or not related to the study intervention.
Outcome measures
| Measure |
Infusions of Young Plasma
n=16 Participants
All participants will undergo neuropsychological, neuropsychiatric, and kinematic assessments prior to receiving infusions of young plasma as the treatment.
Participants will receive 1 unit of young plasma, twice a week over a four week duration.
After the four weeks of plasma infusions, participants will undergo neuropsychological, neuropsychiatric and kinematic reassessments. No deception will be used.
Infusions of young plasma: Participants will receive four twice- weekly infusions of 1 unit young plasma (male, ages between 18-25)
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Immediate-post Evaluation
Immediately after the last plasma infusion
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Delayed-post Evaluation
4-weeks after the last plasma infusion
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|---|---|---|---|
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Number of Related and Unrelated Adverse Events
Number of probably related AEs
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14 events
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—
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—
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Number of Related and Unrelated Adverse Events
Number of possibly related AEs
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3 events
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—
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—
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Number of Related and Unrelated Adverse Events
Number of unrelated AEs
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36 events
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—
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—
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OTHER_PRE_SPECIFIED outcome
Timeframe: 8 weeksPopulation: Participants who completed the protocol are included in the analysis.
Change in quantitative data of cognitive ability. Raw scores and normative scores were calculated for all measures according to standardized procedures across all 3 time points. The cognitive test battery included: * Trail Making Test Part A (0-92; lower scores indicate better performance) and B (0-300; lower scores indicate better performance) * Digit Symbol Modalities Test (Oral: 0-110 and Written: 0-110; higher scores indicate better performance) * Animal Naming Test (0-no maximum; higher scores indicate better performance) * Controlled Oral Word Association Test (COWAT) (0-no maximum; higher score indicates better performance) * CogStateTM Groton Maze Learning Test (GML) (minimum score is 0; lower scores indicate better performance) * Wechsler Abbreviated Scale of Intelligence-II (WASI-II) Block Design (0-71; higher scores indicate better performance) and Matrix Reasoning (0-30; higher scores indicate better performance)
Outcome measures
| Measure |
Infusions of Young Plasma
n=15 Participants
All participants will undergo neuropsychological, neuropsychiatric, and kinematic assessments prior to receiving infusions of young plasma as the treatment.
Participants will receive 1 unit of young plasma, twice a week over a four week duration.
After the four weeks of plasma infusions, participants will undergo neuropsychological, neuropsychiatric and kinematic reassessments. No deception will be used.
Infusions of young plasma: Participants will receive four twice- weekly infusions of 1 unit young plasma (male, ages between 18-25)
|
Immediate-post Evaluation
n=15 Participants
Immediately after the last plasma infusion
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Delayed-post Evaluation
n=15 Participants
4-weeks after the last plasma infusion
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|---|---|---|---|
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Change in Quantitative Data of Cognitive Ability (Neuropsychological Battery)
WASI-II Matrix Reasoning
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17.47 score on a scale
Interval 14.87 to 20.06
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17.27 score on a scale
Interval 14.67 to 19.86
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20.13 score on a scale
Interval 17.54 to 22.73
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Change in Quantitative Data of Cognitive Ability (Neuropsychological Battery)
Trail Making Test-B
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83.20 score on a scale
Interval 62.64 to 103.76
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72.87 score on a scale
Interval 52.31 to 93.42
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73.53 score on a scale
Interval 52.98 to 94.09
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Change in Quantitative Data of Cognitive Ability (Neuropsychological Battery)
Symbol Digit Modalities Test - Written
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44.80 score on a scale
Interval 39.14 to 50.46
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47.07 score on a scale
Interval 41.4 to 52.73
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49.40 score on a scale
Interval 43.74 to 55.06
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Change in Quantitative Data of Cognitive Ability (Neuropsychological Battery)
Symbol Digit Modalities Test - Oral
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53.67 score on a scale
Interval 46.89 to 60.44
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57.40 score on a scale
Interval 50.62 to 64.18
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57.73 score on a scale
Interval 50.96 to 64.51
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Change in Quantitative Data of Cognitive Ability (Neuropsychological Battery)
Trail Making Test-A
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32.53 score on a scale
Interval 27.63 to 37.44
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29.27 score on a scale
Interval 24.36 to 34.17
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29.07 score on a scale
Interval 24.16 to 33.97
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Change in Quantitative Data of Cognitive Ability (Neuropsychological Battery)
Phonemic fluency (COWAT; Letters-FAS)
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43.13 score on a scale
Interval 34.75 to 47.52
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44.73 score on a scale
Interval 38.35 to 51.12
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48.07 score on a scale
Interval 41.68 to 54.45
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Change in Quantitative Data of Cognitive Ability (Neuropsychological Battery)
Semantic fluency (Animal naming)
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21.40 score on a scale
Interval 19.17 to 23.63
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22.40 score on a scale
Interval 20.17 to 24.63
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23.07 score on a scale
Interval 20.84 to 25.29
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Change in Quantitative Data of Cognitive Ability (Neuropsychological Battery)
CogState (GML)
|
71.47 score on a scale
Interval 53.15 to 89.79
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54.87 score on a scale
Interval 36.55 to 73.19
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60.53 score on a scale
Interval 42.21 to 78.85
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Change in Quantitative Data of Cognitive Ability (Neuropsychological Battery)
WASI-II Block Construction
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34.73 score on a scale
Interval 27.45 to 42.02
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35.60 score on a scale
Interval 28.32 to 42.88
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37.47 score on a scale
Interval 30.18 to 44.75
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OTHER_PRE_SPECIFIED outcome
Timeframe: 8 weeksPopulation: Participants who completed the protocol are included in the analysis.
Quality of life (QOL) changes were tracked using the self-report Parkinson's Disease Questionnaire-39 (PDQ-39). The PDQ-39 includes 8 sub-scales: Mobility (raw score range 0-40), Activities of Daily Living (raw score range 0-24), Emotional Well-Being (raw score range 0-24), Stigma (raw score range 0-16), Social Support (raw score range 0-12), Cognition (raw score range 0-16), Communication (raw score range 0-12), and Bodily Discomfort (raw score range 0-12). Subscales scores are totaled then converted to a percentage to calculate the Total score (0 to 100, higher scores indicating the more problems). Ratings are based on participant experiences over the course of the prior month. Lower scores represent better quality of life for all scores.
Outcome measures
| Measure |
Infusions of Young Plasma
n=15 Participants
All participants will undergo neuropsychological, neuropsychiatric, and kinematic assessments prior to receiving infusions of young plasma as the treatment.
Participants will receive 1 unit of young plasma, twice a week over a four week duration.
After the four weeks of plasma infusions, participants will undergo neuropsychological, neuropsychiatric and kinematic reassessments. No deception will be used.
Infusions of young plasma: Participants will receive four twice- weekly infusions of 1 unit young plasma (male, ages between 18-25)
|
Immediate-post Evaluation
n=15 Participants
Immediately after the last plasma infusion
|
Delayed-post Evaluation
n=15 Participants
4-weeks after the last plasma infusion
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|---|---|---|---|
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Change in Quality of Life
Cognition
|
6.90 score on a scale
Interval 5.53 to 8.28
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6.87 score on a scale
Interval 5.5 to 8.23
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6.80 score on a scale
Interval 5.43 to 8.17
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Change in Quality of Life
Communication
|
5.33 score on a scale
Interval 4.31 to 6.36
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5.20 score on a scale
Interval 4.19 to 6.21
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4.60 score on a scale
Interval 3.59 to 5.61
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Change in Quality of Life
Bodily Discomfort
|
6.53 score on a scale
Interval 5.25 to 7.82
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5.27 score on a scale
Interval 4.0 to 6.53
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5.80 score on a scale
Interval 4.53 to 7.07
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|
Change in Quality of Life
Total PDQ-39 Score
|
71.81 score on a scale
Interval 59.51 to 84.1
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66.73 score on a scale
Interval 54.49 to 78.98
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64.40 score on a scale
Interval 52.16 to 76.64
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Change in Quality of Life
Mobility
|
17.33 score on a scale
Interval 13.38 to 21.28
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16.47 score on a scale
Interval 12.53 to 20.41
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15.80 score on a scale
Interval 11.86 to 19.74
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Change in Quality of Life
Activities of Daily Living
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12.05 score on a scale
Interval 10.29 to 13.82
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11.60 score on a scale
Interval 9.85 to 13.35
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10.53 score on a scale
Interval 8.79 to 12.28
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Change in Quality of Life
Emotional Well-Being
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10.60 score on a scale
Interval 7.75 to 13.45
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10.13 score on a scale
Interval 7.3 to 12.97
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10.00 score on a scale
Interval 7.16 to 12.84
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Change in Quality of Life
Stigma
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7.42 score on a scale
Interval 6.22 to 8.63
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6.20 score on a scale
Interval 5.01 to 7.39
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5.53 score on a scale
Interval 4.34 to 6.72
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Change in Quality of Life
Social support
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5.57 score on a scale
Interval 3.99 to 7.16
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5.00 score on a scale
Interval 3.43 to 6.57
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5.33 score on a scale
Interval 3.76 to 6.9
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OTHER_PRE_SPECIFIED outcome
Timeframe: 8 weeksPopulation: Patients who completed the rWFE task at baseline and at both outcome visits and who had analyzable data are included in the analysis.
The second outcome measure is the change in the quantitative data of current motor movement ability. Patients completed a repetitive Wrist Flexion Extension (rWFE) task with both hands, off therapy at the baseline, immediate-post, and delayed-post visits. Patients were instructed to flex and extend their hands at the wrist as quickly as possible after a "Go" command was given and to stop when instructed. This movement was self-paced, lasted 30 seconds, and was measured using wearable sensors attached to the dorsum of each hand (Motus Bioengineering, Inc.). Variables of interest included the mean angular velocity (Vrms), the variability of mean angular velocity, (CV Vrms), and rhythmicity, defined as the regularity of the interstrike interval (CV ISI). MA = more affected; LA = less affected.
Outcome measures
| Measure |
Infusions of Young Plasma
n=12 Participants
All participants will undergo neuropsychological, neuropsychiatric, and kinematic assessments prior to receiving infusions of young plasma as the treatment.
Participants will receive 1 unit of young plasma, twice a week over a four week duration.
After the four weeks of plasma infusions, participants will undergo neuropsychological, neuropsychiatric and kinematic reassessments. No deception will be used.
Infusions of young plasma: Participants will receive four twice- weekly infusions of 1 unit young plasma (male, ages between 18-25)
|
Immediate-post Evaluation
n=12 Participants
Immediately after the last plasma infusion
|
Delayed-post Evaluation
n=12 Participants
4-weeks after the last plasma infusion
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|---|---|---|---|
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Change in Quantitative Data of Motor Movements up to 8 Weeks
LA CV ISI
|
0.09 Degrees/sec
Interval 0.05 to 0.14
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0.09 Degrees/sec
Interval 0.04 to 0.13
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0.10 Degrees/sec
Interval 0.05 to 0.15
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|
Change in Quantitative Data of Motor Movements up to 8 Weeks
MA Vrms
|
305.13 Degrees/sec
Interval 196.72 to 413.54
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339.66 Degrees/sec
Interval 231.24 to 448.07
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312.39 Degrees/sec
Interval 203.98 to 420.81
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|
Change in Quantitative Data of Motor Movements up to 8 Weeks
MA CV Vrms
|
0.29 Degrees/sec
Interval 0.2 to 0.38
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0.19 Degrees/sec
Interval 0.1 to 0.29
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0.26 Degrees/sec
Interval 0.17 to 0.35
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Change in Quantitative Data of Motor Movements up to 8 Weeks
MA CV ISI
|
0.16 Degrees/sec
Interval 0.07 to 0.26
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0.11 Degrees/sec
Interval 0.01 to 0.2
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0.17 Degrees/sec
Interval 0.08 to 0.27
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|
Change in Quantitative Data of Motor Movements up to 8 Weeks
LA Vrms
|
414.47 Degrees/sec
Interval 295.08 to 533.86
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411.26 Degrees/sec
Interval 291.87 to 530.65
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403.00 Degrees/sec
Interval 283.61 to 522.39
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|
Change in Quantitative Data of Motor Movements up to 8 Weeks
LA CV Vrms
|
0.16 Degrees/sec
Interval 0.09 to 0.22
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0.17 Degrees/sec
Interval 0.12 to 0.24
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0.18 Degrees/sec
Interval 0.11 to 0.24
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Adverse Events
Infusions of Young Plasma
Serious events: 0 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Infusions of Young Plasma
n=16 participants at risk
All participants will undergo neuropsychological, neuropsychiatric, and kinematic assessments prior to receiving infusions of young plasma as the treatment.
Participants will receive 1 unit of young plasma, twice a week over a four week duration.
After the four weeks of plasma infusions, participants will undergo neuropsychological, neuropsychiatric and kinematic reassessments. No deception will be used.
Infusions of young plasma: Participants will receive four twice- weekly infusions of 1 unit young plasma (male, ages between 18-25)
|
|---|---|
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Skin and subcutaneous tissue disorders
Bruising
|
6.2%
1/16 • Number of events 1 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorder
|
50.0%
8/16 • Number of events 15 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
12.5%
2/16 • Number of events 2 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
Blood and lymphatic system disorders
Hypotension
|
6.2%
1/16 • Number of events 1 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
Nervous system disorders
Nervous system disturbance
|
18.8%
3/16 • Number of events 4 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
Gastrointestinal disorders
Bloating
|
6.2%
1/16 • Number of events 1 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
Renal and urinary disorders
Urinary frequency distrubance
|
12.5%
2/16 • Number of events 2 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
Gastrointestinal disorders
Stomach pain
|
6.2%
1/16 • Number of events 1 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
6.2%
1/16 • Number of events 1 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder
|
18.8%
3/16 • Number of events 4 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
General disorders
Chest tightness
|
6.2%
1/16 • Number of events 1 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
6.2%
1/16 • Number of events 1 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
General disorders
Fall
|
6.2%
1/16 • Number of events 1 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
General disorders
Flu-like symptoms
|
12.5%
2/16 • Number of events 2 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
General disorders
Tremor
|
6.2%
1/16 • Number of events 1 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
General disorders
Involuntary movement
|
31.2%
5/16 • Number of events 8 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
6.2%
1/16 • Number of events 1 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
General disorders
Sleep disturbance
|
12.5%
2/16 • Number of events 2 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
General disorders
Headache
|
12.5%
2/16 • Number of events 2 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
Gastrointestinal disorders
Gastrointestinal disorders
|
6.2%
1/16 • Number of events 1 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
|
General disorders
Pain in extremity
|
6.2%
1/16 • Number of events 1 • Adverse events were monitored during the entirety of the trial, starting at baseline and continuing through the 4 week post-infusion mark (8 week total).
Adverse events were categorized using the CTCAE v4.0.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place